JP2011521627A5 - - Google Patents
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- JP2011521627A5 JP2011521627A5 JP2011508686A JP2011508686A JP2011521627A5 JP 2011521627 A5 JP2011521627 A5 JP 2011521627A5 JP 2011508686 A JP2011508686 A JP 2011508686A JP 2011508686 A JP2011508686 A JP 2011508686A JP 2011521627 A5 JP2011521627 A5 JP 2011521627A5
- Authority
- JP
- Japan
- Prior art keywords
- fusion protein
- igf
- targeted therapeutic
- therapeutic fusion
- seq
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 108020001507 fusion proteins Proteins 0.000 claims description 54
- 102000037865 fusion proteins Human genes 0.000 claims description 54
- 230000001225 therapeutic effect Effects 0.000 claims description 52
- 102000048143 Insulin-Like Growth Factor II Human genes 0.000 claims description 33
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 claims description 33
- 150000001413 amino acids Chemical class 0.000 claims description 22
- 230000002132 lysosomal effect Effects 0.000 claims description 16
- 102000004190 Enzymes Human genes 0.000 claims description 15
- 108090000790 Enzymes Proteins 0.000 claims description 15
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 14
- 101001076292 Homo sapiens Insulin-like growth factor II Proteins 0.000 claims description 11
- 102000057877 human IGF2 Human genes 0.000 claims description 11
- 238000000034 method Methods 0.000 claims description 11
- 238000006467 substitution reaction Methods 0.000 claims description 11
- 230000037430 deletion Effects 0.000 claims description 10
- 238000012217 deletion Methods 0.000 claims description 10
- 102000004961 Furin Human genes 0.000 claims description 9
- 108090001126 Furin Proteins 0.000 claims description 9
- 230000035772 mutation Effects 0.000 claims description 9
- 238000012258 culturing Methods 0.000 claims description 8
- 102000039446 nucleic acids Human genes 0.000 claims description 8
- 108020004707 nucleic acids Proteins 0.000 claims description 8
- 150000007523 nucleic acids Chemical class 0.000 claims description 8
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 claims description 6
- 101001028831 Homo sapiens Cation-independent mannose-6-phosphate receptor Proteins 0.000 claims description 6
- 108010001127 Insulin Receptor Proteins 0.000 claims description 6
- 208000015439 Lysosomal storage disease Diseases 0.000 claims description 6
- 210000004027 cell Anatomy 0.000 claims description 6
- 102000003746 Insulin Receptor Human genes 0.000 claims description 5
- 238000003776 cleavage reaction Methods 0.000 claims description 5
- 230000007017 scission Effects 0.000 claims description 5
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims description 4
- 108010031794 IGF Type 1 Receptor Proteins 0.000 claims description 4
- 102100039688 Insulin-like growth factor 1 receptor Human genes 0.000 claims description 4
- 239000006143 cell culture medium Substances 0.000 claims description 4
- 230000002950 deficient Effects 0.000 claims description 4
- 102000045921 human GAA Human genes 0.000 claims description 4
- 210000004962 mammalian cell Anatomy 0.000 claims description 4
- 125000006850 spacer group Chemical group 0.000 claims description 4
- QXPRJQPCFXMCIY-NKWVEPMBSA-N Gly-Ala-Pro Chemical compound C[C@@H](C(=O)N1CCC[C@@H]1C(=O)O)NC(=O)CN QXPRJQPCFXMCIY-NKWVEPMBSA-N 0.000 claims description 2
- 102220576120 Oligodendrocyte transcription factor 1_Y27W_mutation Human genes 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 102200149451 rs1800361 Human genes 0.000 claims description 2
- 102220060032 rs772591447 Human genes 0.000 claims description 2
- 102100034561 Alpha-N-acetylglucosaminidase Human genes 0.000 claims 2
- 102100026189 Beta-galactosidase Human genes 0.000 claims 2
- 102000004882 Lipase Human genes 0.000 claims 2
- 108090001060 Lipase Proteins 0.000 claims 2
- 108010009380 alpha-N-acetyl-D-glucosaminidase Proteins 0.000 claims 2
- 108010005774 beta-Galactosidase Proteins 0.000 claims 2
- 102100031317 Alpha-N-acetylgalactosaminidase Human genes 0.000 claims 1
- 102100022146 Arylsulfatase A Human genes 0.000 claims 1
- 102100031491 Arylsulfatase B Human genes 0.000 claims 1
- 101710124976 Beta-hexosaminidase A Proteins 0.000 claims 1
- 101710124978 Beta-hexosaminidase B Proteins 0.000 claims 1
- 102100032487 Beta-mannosidase Human genes 0.000 claims 1
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 claims 1
- 102100028496 Galactocerebrosidase Human genes 0.000 claims 1
- 108010042681 Galactosylceramidase Proteins 0.000 claims 1
- IAJILQKETJEXLJ-UHFFFAOYSA-N Galacturonsaeure Natural products O=CC(O)C(O)C(O)C(O)C(O)=O IAJILQKETJEXLJ-UHFFFAOYSA-N 0.000 claims 1
- 102000004547 Glucosylceramidase Human genes 0.000 claims 1
- 108010017544 Glucosylceramidase Proteins 0.000 claims 1
- 102000053187 Glucuronidase Human genes 0.000 claims 1
- 108010060309 Glucuronidase Proteins 0.000 claims 1
- 108010000540 Hexosaminidases Proteins 0.000 claims 1
- 102000002268 Hexosaminidases Human genes 0.000 claims 1
- 102000004627 Iduronidase Human genes 0.000 claims 1
- 108010003381 Iduronidase Proteins 0.000 claims 1
- 102100036721 Insulin receptor Human genes 0.000 claims 1
- AEMOLEFTQBMNLQ-HNFCZKTMSA-N L-idopyranuronic acid Chemical compound OC1O[C@@H](C(O)=O)[C@@H](O)[C@H](O)[C@H]1O AEMOLEFTQBMNLQ-HNFCZKTMSA-N 0.000 claims 1
- 108010027520 N-Acetylgalactosamine-4-Sulfatase Proteins 0.000 claims 1
- 102100023282 N-acetylglucosamine-6-sulfatase Human genes 0.000 claims 1
- 108010023320 N-acetylglucosamine-6-sulfatase Proteins 0.000 claims 1
- 101000652829 Neosartorya fumigata (strain ATCC MYA-4609 / Af293 / CBS 101355 / FGSC A1100) Exo-alpha-sialidase Proteins 0.000 claims 1
- 102000005327 Palmitoyl protein thioesterase Human genes 0.000 claims 1
- 108020002591 Palmitoyl protein thioesterase Proteins 0.000 claims 1
- 102000035195 Peptidases Human genes 0.000 claims 1
- 108091005804 Peptidases Proteins 0.000 claims 1
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 1
- 102000005262 Sulfatase Human genes 0.000 claims 1
- 108010044965 UDP-N-acetylglucosamine-lysosomal-enzyme N-acetylglucosaminephosphotransferase Proteins 0.000 claims 1
- 102000005421 acetyltransferase Human genes 0.000 claims 1
- 108020002494 acetyltransferase Proteins 0.000 claims 1
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 claims 1
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 1
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 1
- 102000012086 alpha-L-Fucosidase Human genes 0.000 claims 1
- 108010061314 alpha-L-Fucosidase Proteins 0.000 claims 1
- 108010012864 alpha-Mannosidase Proteins 0.000 claims 1
- 102000019199 alpha-Mannosidase Human genes 0.000 claims 1
- 108010015684 alpha-N-Acetylgalactosaminidase Proteins 0.000 claims 1
- 108010055059 beta-Mannosidase Proteins 0.000 claims 1
- 108010089932 heparan sulfate sulfatase Proteins 0.000 claims 1
- 235000019833 protease Nutrition 0.000 claims 1
- 230000001681 protective effect Effects 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 108060007951 sulfatase Proteins 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 description 1
- 208000015872 Gaucher disease Diseases 0.000 description 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 description 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 description 1
- 125000000539 amino acid group Chemical group 0.000 description 1
- 201000004502 glycogen storage disease II Diseases 0.000 description 1
- 230000037431 insertion Effects 0.000 description 1
- 238000003780 insertion Methods 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US5133608P | 2008-05-07 | 2008-05-07 | |
| US61/051,336 | 2008-05-07 | ||
| US14410609P | 2009-01-12 | 2009-01-12 | |
| US61/144,106 | 2009-01-12 | ||
| PCT/US2009/043207 WO2009137721A2 (en) | 2008-05-07 | 2009-05-07 | Lysosomal targeting peptides and uses thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2011521627A JP2011521627A (ja) | 2011-07-28 |
| JP2011521627A5 true JP2011521627A5 (https=) | 2013-05-23 |
| JP5627571B2 JP5627571B2 (ja) | 2014-11-19 |
Family
ID=41265422
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011508686A Active JP5627571B2 (ja) | 2008-05-07 | 2009-05-07 | リソソーム標的化ペプチドおよびその使用 |
Country Status (16)
| Country | Link |
|---|---|
| US (8) | US20110223147A1 (https=) |
| EP (4) | EP3272773B8 (https=) |
| JP (1) | JP5627571B2 (https=) |
| CN (1) | CN102066422B (https=) |
| AU (1) | AU2009244148B2 (https=) |
| BR (2) | BRPI0912225A2 (https=) |
| CA (1) | CA2723412A1 (https=) |
| CY (1) | CY1119183T1 (https=) |
| DK (1) | DK2279210T3 (https=) |
| ES (2) | ES2830350T3 (https=) |
| HR (1) | HRP20170698T1 (https=) |
| HU (1) | HUE034850T2 (https=) |
| IL (1) | IL209091A (https=) |
| PL (1) | PL2279210T3 (https=) |
| PT (1) | PT2279210T (https=) |
| WO (1) | WO2009137721A2 (https=) |
Families Citing this family (62)
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| CA2887752C (en) | 2007-04-03 | 2020-03-24 | Vib Vzw | Glycosylation of molecules |
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| JP5627571B2 (ja) | 2008-05-07 | 2014-11-19 | バイオマリン ファーマシューティカル インコーポレイテッド | リソソーム標的化ペプチドおよびその使用 |
| US8540985B2 (en) | 2008-06-26 | 2013-09-24 | Orphazyme Aps | Use of Hsp70 as a regulator of enzymatic activity |
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| EP2485761B1 (en) | 2009-10-09 | 2019-02-27 | Armagen, Inc. | Methods and compositions for increasing iduronate 2-sulfatase activity in the cns |
| WO2011061629A2 (en) | 2009-11-19 | 2011-05-26 | Oxyrane Uk Limited | Yeast strains producing mammalian-like complex n-glycans |
| HRP20191923T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| AU2011270672B2 (en) * | 2010-06-25 | 2017-01-12 | Shire Human Genetic Therapies, Inc. | Treatment of Sanfilippo Syndrome type B |
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| KR20230159646A (ko) | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物 |
| US9347050B2 (en) | 2010-09-29 | 2016-05-24 | Oxyrane Uk Limited | Mannosidases capable of uncapping mannose-1-phospho-6-mannose linkages and demannosylating phosphorylated N-glycans and methods of facilitating mammalian cellular uptake of glycoproteins |
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| CN102121021B (zh) * | 2010-12-21 | 2012-05-02 | 南京师范大学 | 绵羊IFN-γ诱导的溶酶体巯基还原酶cDNA及其克隆方法和重组应用 |
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